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BackgroundFew studies assessing the correlation between patient-reported outcomes and patient-generated health data from wearable devices exist. ObjectiveThe aim of this study was to determine the direction and magnitude of associations between patient-generated health data (from the Fitbit Charge HR) and patient-reported outcomes for sleep patterns and physical activity in patients with type 2 diabetes mellitus (T2DM). MethodsThis was a pilot study conducted with adults diagnosed with T2DM (n=86). All participants wore a Fitbit Charge HR for 14 consecutive days and completed internet-based surveys at 3 time points: day 1, day 7, and day 14. Patient-generated health data included minutes asleep and number of steps taken. Questionnaires assessed the number of days of exercise and nights of sleep problems per week. Means and SDs were calculated for all data, and Pearson correlations were used to examine associations between patient-reported outcomes and patient-generated health data. All respondents provided informed consent before participating. ResultsThe participants were predominantly middle-aged (mean 54.3, SD 13.3 years), white (80/86, 93%), and female (50/86, 58%). Use of oral T2DM medication correlated with the number of mean steps taken (r=.35, P=.001), whereas being unaware of the glycated hemoglobin level correlated with the number of minutes asleep (r=−.24, P=.04). On the basis of the Fitbit data, participants walked an average of 4955 steps and slept 6.7 hours per day. They self-reported an average of 2.0 days of exercise and 2.3 nights of sleep problems per week. The association between the number of days exercised and steps walked was strong (r=.60, P

The small arginine-rich protein protamine condenses complete genomic DNA into the sperm head. Here, we applied its high RNA binding capacity for spontaneous electrostatic assembly of therapeutic nanoparticles decorated with tumour-cell-specific antibodies for efficiently targeting siRNA. Fluorescence microscopy and DLS measurements of these nanocarriers revealed the formation of a vesicular architecture that requires presence of antibody-protamine, defined excess of free SMCC-protamine, and anionic siRNA to form. Only these complex nanoparticles were efficient in the treatment of non-small-cell lung cancer (NSCLC) xenograft models, when the oncogene KRAS was targeted via EGFR-mediated delivery. To show general applicability, we used the modular platform for IGF1R-positive Ewing sarcomas. Anti-IGR1R-antibodies were integrated into an antibody-protamine nanoparticle with an siRNA specifically against the oncogenic translocation product EWS/FLI1. Using these nanoparticles, EWS/FLI1 knockdown blocked in vitro and in vivo growth of Ewing sarcoma cells. We conclude that these antibody-protamine-siRNA nanocarriers provide a novel platform technology to specifically target different cell types and yet undruggable targets in cancer therapy by RNAi.

Disorders/differences of sex development (DSD) are congenital conditions in which there is atypical chromosomal, gonadal and/or phenotypic sex. While there remains controversy around the traditionally binary concept of sex, most patients with DSD are reared either male or female depending on their genetic sex, gonadal sex, genital phenotype and status of their internal genital tract. This study uses prospective data from 12 institutions across the United States that specialize in DSD care. We focused on patients raised female. Eligible patients had moderate to severe genital atypia (defined as Prader score >2), were ≤2 years of age at entry, and had no prior genitoplasty. The aim of this study is to describe early post operative complications for young patients undergoing modern approaches to feminizing genitoplasty. Of the 91 participants in the cohort, 57 (62%) were reared female. The majority had congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (n = 52), 1 had ovo-testicular syndrome, 2 had mixed gonadal dysgenesis and 2 had partial androgen insensitivity syndrome (PAIS). Of the 50 participants who received early genitoplasty, 43 (86%) had follow-up at 6-12 months post-surgery. Thirty-two participants (64%) received a clitoroplasty, 31 (62%) partial urogenital mobilization and 4 (8%) total urogenital sinus mobilization. Eighteen percent (9/50) experienced post-surgical complications with 7 (14%) being rated as Clavien-Dindo grade III. Both parents and surgeons reported improved satisfaction with genital appearance of participants following surgery compared to baseline. This information on post-operative complications associated with contemporary approaches to feminizing genitoplasty performed in young children will help guide families when making decisions about whether or not to proceed with surgery for female patients with moderate to severe genital atypia.

PurposeWe examined the psychosocial characteristics of parents of children with disorders of sex development at early presentation to a disorders of sex development clinic. Parental anxiety, depression, quality of life, illness uncertainty and posttraumatic stress symptoms were assessed. Additionally we evaluated the relationship of assigned child gender to parental outcomes.Materials and methodsA total of 51 parents of children with ambiguous or atypical genitalia were recruited from 7 centers specializing in treatment of disorders of sex development. At initial assessment no child had undergone genitoplasty. Parents completed the Cosmetic Appearance Rating Scale, Beck Anxiety Inventory, Beck Depression Inventory, SF-36, Parent Perception of Uncertainty Scale and Impact of Event Scale-Revised.ResultsA large percentage of parents (54.5%) were dissatisfied with the genital appearance of their child, and a small but significant percentage reported symptoms of anxiety, depression, diminished quality of life, uncertainty and posttraumatic stress. Few gender differences emerged.ConclusionsAlthough many parents function well, a subset experience significant psychological distress around the time of diagnosis of a disorder of sex development in their child. Early screening to assess the need for psychosocial interventions is warranted.

Aim: To assess perspectives on clinical communication about sexual health in young adult males with spina bifida. Method: Semi‐structured interviews were conducted between February and May 2021 with males at least 18 years of age with spina bifida to assess their perspectives and experiences of sexual health communication with clinicians. Demographic and clinical characteristics were obtained from chart review and a survey of patients. Interviews were transcribed verbatim, and conventional content analysis was used for transcript coding. Results: Twenty individuals participated with a median age of 22 years 6 months (range 18–29 years). Sixteen had myelomeningocele. Most identified as heterosexual (n = 17) and not sexually active (n = 13). Barriers and facilitators of successful interactions were identified. Barriers for participants included general discomfort with talking about sex and variability in individual preferences for how conversations occur. Facilitators included participants' comfort with their urologist and discussing sex in relation to disability. Suggestions for improving discussions included (1) notifying individuals that discussion about sex will occur before clinic visits; (2) creating space for discussions; (3) respecting individuals' readiness to discuss; and (4) making discussions disability specific. Interpretation: Young adult males with spina bifida are interested in discussing sexual health with their clinicians. Great variability exists about conversation preferences, emphasizing the need to individualize clinical communication about sex. Current health guidelines for males may not be in line with individuals' wishes. What this paper adds: Great variability exists in individual preference around sexual health communication.Patient‐level barriers hinder successful conversations about sex.Individuals have great insight into how conversations about sex can be improved. [ABSTRACT FROM AUTHOR]